Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)

Launched by HAIHE BIOPHARMA CO., LTD. · May 13, 2025

Keywords

ClinConnect Summary

This clinical trial is testing a new medication called CYH33 for patients with certain conditions known as PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM). The main goals of the study are to see how safe the medication is, how well the body absorbs it, and whether it shows any early signs of helping patients. The trial is currently looking for participants, and it welcomes individuals aged 12 years and older, including adults. To join, patients must be diagnosed with one of these conditions and have good organ function.

Participants in this trial will receive the medication and will be monitored closely for any side effects or improvements in their condition. It's important to note that individuals who have previously used certain treatments for their condition or who have specific health issues, like serious heart disease, may not be eligible to participate. This study aims to gather valuable information that could help improve treatment options for patients with these conditions in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • The patient or the patient's guardian (if applicable) voluntarily signs the Informed Consent Form.
• • At the time of signing the Informed Consent, the age of adult patients should be ≥ 18 years, and the age of adolescent patients should be ≥ 12 years and \< 18 years of age.
• • Patient diagnosed with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformation (PRVM).
• • Patients should be tested to have adequate organ and bone marrow function, within 28 days of the screening period.
• Exclusion Criteria:
• • Patients who have received any prior treatment with PI3K or AKT inhibitors.
• • Received a blood transfusion or platelet transfusion within 2 weeks prior to the administration of the investigational drug.
• • Patients with clinically significant cardiovascular disease.